MSCA-DN PhD in lipid nanoparticle gene therapy for leukodystrophies (London, UK)
POLARIS doctoral network
POLARIS is a European Marie Skłodowska-Curie Doctoral Network focused on accelerating therapy development for leukodystrophies: rare genetic disorders affecting the brain's white matter. The network aims to strengthen the full translational pathway, from understanding disease mechanisms and developing disease models to preclinical therapy testing, clinical readiness and patient-informed treatment priorities.
To achieve this, POLARIS brings together expertise in leukodystrophy biology, advanced disease models, therapeutic innovation, biomarker development, clinical translation, patient engagement, and ethical, regulatory and health-economic aspects of therapy development. The network will train a cohort of doctoral candidates in an international and interdisciplinary environment, combining academic research, industry-based preclinical development, secondments, network-wide training and transferable-skills development.
PhD project
For this position, University College London (UCL) is the recruiting organisation. You will be employed by UCL and embedded in the POLARIS consortium. Your academic supervision and disease-specific expertise will be provided by Professor Ahad A. Rahim (UCL School of Pharmacy), Professor Stephen Hart and Dr Julien Baruteau (UCL Great Ormond Street Institute of Child Health).
Your PhD project focuses on the in vitro and in vivo preclinical assessment of genetic therapies for leukodystrophies. You will investigate lipid nanoparticle (LNP)-based approaches for delivering therapeutic nucleic acids, such as mRNA, in cellular and animal models.
The research follows a translational pathway towards clinical application. You will investigate the functionality, biodistribution and cellular tropism of LNP-mRNA delivery systems and evaluate their therapeutic efficacy in mouse models of leukodystrophies.
A planned secondment at the University of York will provide experience of working with patient-family organisations and exposure to additional disease models.
As a doctoral candidate, you will work across different stages of the preclinical research process. Your activities include:
- Testing the functionality of LNP-mRNA delivery systems in vitro.
- Analysing the biodistribution of delivery systems in the brain and peripheral tissues and investigating cellular tropism following different administration routes in mice.
- Conducting a preclinical study using the most promising LNP-mRNA delivery system and administration route in mouse models of leukodystrophies.
- Evaluating therapeutic efficacy using neuropathology, lifespan, locomotor and behavioural studies.
- Working with translational research supervisors and POLARIS partners to move promising approaches towards clinical application.
We are looking for a candidate with a Master's degree in life sciences and relevant research experience. You bring the following:
- Experience with molecular technologies, neuroscience and in vivo mouse studies, gained through internships, thesis work or research projects.
- A strong interest in translational neuroscience, rare disease research and the development of genetic therapies.
- Experience with LNPs and animal models of neurological disease is an advantage.
- An accurate and reproducible approach to research, with strong attention to detail and the ability to plan and organise complex experimental work.
- Good analytical, communication and collaboration skills and the ability to work effectively in an interdisciplinary and international environment.
Fluency in spoken and written English is essential for this international research environment.
Eligibility criteria
Important, strict eligibility criteria according to MARIE SKLODOWSKA CURIE programs apply to applicants:
- You must not already hold a doctoral degree.
- At the time of the application deadline, you must have a Master's degree and must not have resided or carried out your main activity (work, studies, etc.) in the UK for more than 12 months in the three years prior to the contract start date.
- You will participate in the network-wide training programme of POLARIS, which includes (international) training activities and planned secondments with POLARIS partner organizations throughout the EU.
A contract for a total of 3 years;
Salary, mobility allowance and benefits according to the applicable MSCA Doctoral Network and host-organisation rules. More detailed information about the salary will follow;
Embedding in the international POLARIS doctoral training network;
Access to network-wide training activities, secondments and transferable-skills development;
A challenging and translational research project at the interface of in vivo neuroscience, rare disease biology and preclinical genetic therapy development;
A collaborative and entrepreneurial working environment;
An open and supportive team culture with opportunities for scientific and professional development.
You will work at University College London (UCL), within the UCL School of Pharmacy and the UCL Great Ormond Street Institute of Child Health.
UCL is ranked among the world's leading universities. The UCL School of Pharmacy is part of the Faculty of Life Sciences and has 180 years of experience in research and education. The School is internationally recognised for its research in pharmacy and pharmacology.
Professor Rahim's laboratory has extensive experience in genetic therapies and nucleic acid delivery technologies for rare paediatric neurodegenerative diseases. At the UCL Great Ormond Street Institute of Child Health, researchers work closely with Great Ormond Street Hospital. The institute is one of Europe's largest centres for children's health research. Professor Hart specialises in non-viral nucleic acid delivery systems, while Dr Baruteau combines clinical expertise with translational research.
You will also be part of the international POLARIS doctoral network. You will work with researchers from different scientific disciplines and collaborate with academic and industry partners across Europe. Through secondments, network-wide training and transferable-skills development, you will build experience beyond your own research project and develop an international research network.
POLARIS is committed to open, transparent and merit-based recruitment. We value diversity and welcome candidates with different backgrounds, perspectives and experiences.
The application deadline is 5 October 2026. We aim to select the final candidate in October/November 2026.
Applicants may, where appropriate and in line with the recruitment procedure, also be considered for other POLARIS doctoral candidate positions that better match their profile and interests. You can highlight up to two additional POLARIS projects of interest in your application letter. This does not replace your application to the primary position, but helps the consortium identify potential alternative matches where relevant.
Would you like to know more about this PhD position? For substantive information about the vacancy, please contact Professor Ahad Rahim, via a.rahim@ucl.ac.uk.
Acquisition in response to this vacancy will not be considered.
